Splice-switching antisense oligonucleotides correct cryptic exon inclusion and restore SDCCAG8 protein in Bardet-Biedl Syndrome
剪接转换反义寡核苷酸可纠正隐蔽外显子插入并恢复巴德-比德尔综合征中的 SDCCAG8 蛋白
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doi:10.1101/2025.10.15.682674
McEntee, Kelleen E; McCurdy, Bailey L; Larson, Austin; McCourt, Emily A; Kaufman, Michael L; Campbell, Amy E; Pearson, Chad G; Demarest, Scott; Taliaferro, J Matthew; Hesselberth, Jay R; Jagannathan, Sujatha
